RST-001 Phase I/II Trial for Advanced Retinitis Pigmentosa

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT02556736
Recruitment Status : Recruiting
First Posted : September 22, 2015
Last Update Posted : August 22, 2018
Information provided by (Responsible Party):

September 21, 2015
September 22, 2015
August 22, 2018
December 14, 2015
August 28, 2019   (Final data collection date for primary outcome measure)
Safety [ Time Frame: 6 month from start of study treatment ]
Absence of any grade 3 or greater AE considered related to RST-001.
Safety [ Time Frame: 6 months ]
Any Grade 3 (National Cancer Institute [NCI] grading system) or greater adverse event considered related to injected test article
Complete list of historical versions of study NCT02556736 on Archive Site
  • To establish the maximum tolerated dose of RST-001 [ Time Frame: Up to 24 months from start of study treatment ]
    Determined by a safety review
  • To evaluate the preliminary efficacy of RST-001 in patients with advanced RP [ Time Frame: Up to 24 months from start of study treatment ]
    Preliminary efficacy data will be obtained through ocular evaluations (e.g., number of participants with changes in visual function)
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RST-001 Phase I/II Trial for Advanced Retinitis Pigmentosa
Phase I/IIa, Open-Label, Dose-Escalation Study of Safety and Tolerability of Intravitreal RST-001 in Patients With Advanced Retinitis Pigmentosa (RP)
RST-001 is a gene therapy given as an injection into the eye.
Not Provided
Phase 1
Phase 2
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Advanced Retinitis Pigmentosa
Drug: RST-001
RST-001 is a gene therapeutic delivered by intravitreal injection
Experimental: Group 1
Single intravitreal injection of RST-001
Intervention: Drug: RST-001
Simunovic MP, Shen W, Lin JY, Protti DA, Lisowski L, Gillies MC. Optogenetic approaches to vision restoration. Exp Eye Res. 2018 Sep 13;178:15-26. doi: 10.1016/j.exer.2018.09.003. [Epub ahead of print] Review.

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
October 31, 2033
August 28, 2019   (Final data collection date for primary outcome measure)

Inclusion criteria:

Participants must meet all of the following criteria.

  1. Age >= 18 years
  2. Signed and dated written informed consent obtained from the patient.
  3. Ability to comply with testing and all protocol tests.

Exclusion criteria:

Any one of the following will exclude patients from being enrolled into the study:

  1. Unable or unwilling to meet requirements of the study;
  2. Participation in a clinical study (ocular or non-ocular) with an investigational drug, agent or therapy in the past six months
Sexes Eligible for Study: All
18 Years and older   (Adult, Older Adult)
Contact: Clinical Trials Registry Team 877-277-8566
United States
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Study Director: Werner Schmidt Allergan
August 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP